* This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm. * Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly for 22 weeks (total of 24 weeks of treatment).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
15
Cabaletta for IV infusion once weekly
Meir Medical Center
Kfar Saba, Israel
Adverse events
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time frame: 28 weeks
Physical examination
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time frame: 28 weeks
Vital signs
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time frame: 28weeks
12-lead ECG
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time frame: 28weeks
Safety laboratory tests
Safety will be evaluated on the basis of the following assessments: Adverse events , physical examination, 12-lead ECG, vital signs, safety laboratory evaluations
Time frame: 28weeks
Disease markers
Changes in disease markers will be assessed based on the following assessments: Scale for the Assessment and Rating of Ataxia (SARA); Neurological Examination Score for Spinocerebellar Ataxia (NESSCA); Change in BMI - screening, spinocerebellar Ataxia Functional Tests; quality of life
Time frame: 27 weeks
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